World's First | Puzhong Discovery’s MUC18 ADC Granted Breakthrough Therapy DesignationAttention Index:★★★★★
On December 9, the CDE website announced that Puzhong Discovery Pharmaceutical Technology (Shanghai) Co., Ltd. developedWorld's FirstMUC18 ADC DrugAMT-253Proposed for inclusion in the breakthrough therapy designation, intended for unresectable locally advanced, recurrent, or metastatic melanoma previously treated with first-line therapy. Currently, immune checkpoint inhibitors are the mainstay of first-line treatment for this disease; however, approximately 70% of patients develop resistance, and second-line treatment options are scarce and have limited efficacy.AsThe First in the World to Enter Clinical TrialsMUC18-Targeting ADC, AMT-253: Innovative Target MUC18 Widely Expressed Across Melanoma Subtypes Enables Dual Targeting of Tumor Cells and Tumor Vasculature. Clinical data shows an ORR of 28.6% in heavily pretreated patients without MUC18 screening, with an ORR as high as 50% in acral melanoma and 33.3% in mucosal melanoma, significantly outperforming existing second-line therapies. More advantageously, its exatecan payload is not a substrate for drug resistance efflux pumps, circumventing chemotherapy resistance while showing no severe interstitial pneumonia or neurotoxicity. With its unique target and superior efficacy across subtypes, AMT-253 is well-positioned to capture key markets. Based on the market performance of similar ADC drugs and considering the global annual incidence of over 300,000 new melanoma cases, its peak sales are projected to exceed $1 billion.First in China! Qilu Pharmaceutical's "Panitumumab Biosimilar" Approved for Marketing
Attention Index:★★★★
On December 9, the NMPA website showed that Qilu Pharmaceutical Co., Ltd.Panitumumab Biosimilar (QL1203)Approved for marketing, used in combination with mFOLFOX as first-line treatment for RAS wild-type metastatic colorectal cancer (mCRC) patients. This isChina's First Approved Panitumumab Biosimilar, filling the gap in this treatment field in China.Colorectal cancer is a common malignant tumor in China, with RAS wild-type patients accounting for about half of the cases, representing significant unmet clinical needs. Compared to chemotherapy alone (placebo + mFOLFOX6), the QL1203 combination chemotherapy regimen significantly improved progression-free survival (PFS) in patients, extending the median PFS from 8.34 months to 11.20 months and reducing the risk of disease progression by 39%. China reports 517,100 new cases of colorectal cancer annually, including approximately 200,000 patients with RAS wild-type metastatic colorectal cancer (mCRC), indicating substantial unmet needs. Referencing the global sales of the original cetuximab at €1.025 billion in 2023, QL1203, as the first domestically produced drug of its kind in China, may offer a more affordable price. With the advancement of inclusion in medical insurance, it is projected that within 3-5 years after launch, annual sales could exceed 2 billion yuan, making it an important product in the field of targeted therapy for colorectal cancer.Global Drug Approvals/R&D UpdatesGlobal New Drug ApprovalsAccording to the PharmSnap data analysis, during this statistical period (December 6, 2025 - December 12, 2025), there were a total of 8 new drug approval status updates globally (excluding China). Among them, there were 3 NDA approvals, 1 BLA approval, 3 new indication approvals, and 1 new formulation approval. Compared with the previous statistical period, this period saw an increase of 4 globally approved drugs.
On December 10, Fondazione Telethon announced that the FDA had approved Waskyra (Etuvetidigene autotemcel) for marketing to treat patients aged six months and above with Wiskott-Aldrich Syndrome (WAS) who carry a WAS gene mutation. Waskyra is suitable for those who are eligible for hematopoietic stem cell transplantation (HSCT) but cannot find a suitable donor. This is the first gene therapy approved by the FDA for treating patients with Wiskott-Aldrich Syndrome. Waskyra requires only a single administration via intravenous infusion.The safety and efficacy of Waskyra are based on the results of two open-label, single-arm, multinational clinical studies and an expanded access program, which included a total of 27 patients with severe WAS. The study results showed that for patients with severe WAS, Waskyra can provide significant and lasting clinical benefits and plays an important role in significantly reducing the major disease manifestations that cause morbidity and mortality. Compared with the 12 months prior to treatment, the incidence of severe infections in patients dropped by 93% during the 6 to 18 months post-treatment. Additionally, moderate and severe bleeding events decreased by 60% in the first 12 months after treatment compared to the year before treatment. In follow-ups four years after treatment, most patients no longer reported moderate to severe bleeding events. This approval brings a new treatment option to WAS patients with high unmet medical needs and represents an important milestone in the field of WAS treatment.On December 11, GSK announced that the FDA had approved the oral antibiotic Blujepa (Gepotidacin) for the treatment of uncomplicated urogenital gonorrhea in adolescents and adults aged 12 years and older, caused by drug-susceptible strains of Neisseria gonorrhoeae. The patients must weigh at least 45 kilograms and be unsuitable for, intolerant of, or unwilling to accept standard first-line treatment options. Blujepa is the first new class of antibiotic approved for the treatment of gonorrhea in over 30 years. It exerts antibacterial activity against most target pathogens, including strains resistant to existing antibiotics, by evenly inhibiting two different type II topoisomerases.The marketing application of Blujepa is primarily based on the positive results from the Phase III clinical trial, EAGLE-1. The analysis showed that the urogenital cure rate for patients receiving two oral doses of Blujepa was 92.6% (187/202, 95% CI: 88.0-95.8), demonstrating non-inferiority compared to the cure rate of 91.2% (186/204, 95% CI: 86.4-94.7) achieved with intramuscular Ceftriaxone combined with oral Azithromycin. Neither group experienced urogenital treatment failure due to persistent Neisseria gonorrhoeae, and the safety profile was consistent with previous clinical findings. No serious adverse events related to the drugs were observed, with common adverse reactions being mild to moderate gastrointestinal effects.New Drug Approvals Worldwide (Excluding China) (Partial)
Global New Drug Submission ProgressAccording to the PharmaBlock data analysis, during this statistical period (December 6-12, 2025), there were a total of 7 new drug application progress updates globally (excluding China). Among them, there was 1 chemical drug application progress update, 5 biologic drug application progress updates, and 1 vaccine application progress update. Compared with the previous statistical period, this period saw an increase of 5 NDA/BLA application progress updates.
On December 12, GSK announced that the EMA's Committee for Medicinal Products for Human Use had recommended the approval of Nucala (Mepolizumab) as an add-on maintenance treatment for adult patients with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by elevated eosinophils. Trials demonstrated a statistically significant reduction in the annual rate of moderate to severe exacerbations in the Nucala treatment group when added to triple inhaled therapy, with an annual exacerbation rate of 0.80 in the Nucala group compared to 1.01 in the placebo group.NDA/BLA Submission (Partial)According to the PharmaBlock data statistics analysis, during this statistical period (December 6-12, 2025), a total of 7 drugs worldwide (excluding China) received special regulatory designations. Among them, there were 3 chemical drugs, 1 biologic drug, and 3 cell and gene therapies. Compared with the previous statistical period, there was an increase of 1 drug receiving special regulatory designation in this period.
On December 11, Beren Therapeutics announced that its subsidiary Mandos's investigational drug Adrabetadex has been granted Breakthrough Therapy Designation (BTD) by the U.S. FDA for the treatment of Niemann-Pick disease type C (NPC) with infantile onset. Adrabetadex is a proprietary mixture composed of 2-hydroxypropyl-β-cyclodextrin isomers and is currently being studied in clinical trials for NPC treatment. By restoring intracellular cholesterol transport, adrabetadex directly targets the underlying pathological mechanism of NPC. Existing research results show that adrabetadex is generally well tolerated.On December 11, Atossa Therapeutics announced that the FDA has granted Endoxifen the Rare Pediatric Disease (RPD) designation for the treatment of Duchenne Muscular Dystrophy (DMD). Duchenne Muscular Dystrophy is one of the most severe childhood diseases, and patient families are in urgent need of better treatment options beyond steroid therapies and gene-targeted therapies. Endoxifen, as a potent Selective Estrogen Receptor Modulator/Downregulator (SERM/D), has demonstrated promising potential in preliminary preclinical data through its unique mechanism of action. Since Endoxifen does not target specific exon defects, it is expected to provide a broader and more accessible treatment option for this patient population.Drugs Granted Special Status by Regulatory Authorities Worldwide (Partial)
Global New Drug Development ProgressAccording to the PharmCube data statistics analysis, during this statistical period (December 6-12, 2025), there were a total of 103 updates on the clinical R&D status of new drugs globally (excluding China), involving oncology, neurological diseases, infections, and the cardiovascular system.A total of 16 fields, including diseases.Among them, the clinical progress updates in the field of blood and lymphatic diseases ranked first among all fields, with 42 updates, including 17 chemical drugs, 12 biologics, 12 cell therapies, and 1 vaccine.On December 6, Legend Biotech announced the long-term follow-up clinical and translational research data from the CARTITUDE-1 and CARTITUDE-4 studies of CARVYKTI® (Ciltacabtagene Autoleucel, Cilta-cel) in patients with relapsed or refractory multiple myeloma (RRMM). In patients previously treated with three lines of therapy and triple-exposed, a median progression-free survival (mPFS) of up to 50.4 months was observed after a single infusion of CARVYKTI®. This represents one of the longest progression-free survival (PFS) periods reported for BCMA-targeted CAR-T cell therapy in this heavily treated population.On December 12, AC Immune announced positive interim safety and efficacy results from the Phase II VacSYn trial of its proprietary anti-alpha-synuclein (a-syn) immunotherapy, ACI-7104.056, in patients with early Parkinson's disease (PD). The trial results showed that the therapy met the pre-specified immunogenicity endpoint, with a 100% responder rate among participants. Additionally, it achieved the study-defined goals in pharmacodynamic effects, target engagement, and multiple clinical assessment metrics. In this interim analysis, ACI-7104.056 also demonstrated clear and favorable safety characteristics, with no clinically significant safety issues identified.Global New Drug Development Progress Details (Partial)Global Pharmaceutical Transaction EventsThis statistical cycle（2025.12.06-12.12）There were a total of 41 pharmaceutical transaction events globally (including China), involving multiple transactions such as drug rights transfers and company acquisitions.
Summary Table of Global Pharmaceutical Transaction Times (Partial)
Drug Approval/Development Updates in ChinaNew Drug Approvals in ChinaAccording to the statistical analysis by PharmaDJ, the current statistical cycle (2025.12.06-12.12)In China, a total of 12 new drugs were approved for marketing by the NMPA, including 7 approved through NDAs, 1 approved through a BLA, and 4 new indications. Compared to the previous statistical cycle, this round saw an increase of 11 newly approved drugs by the NMPA.
On December 10, Boehringer Ingelheim announced that the NMPA had approved namilist for the treatment of adult progressive pulmonary fibrosis (PPF). Study data showed that namilist achieved its primary endpoint, which was the absolute change in forced vital capacity (FVC) [mL] from baseline at week 52 compared to the placebo group. FVC is a key indicator of lung function, and the study results indicated that the drug effectively slowed the decline in lung function in PPF patients. Additionally, namilist demonstrated good safety and tolerability, with a low discontinuation rate and no need for liver function monitoring. The FIBRONEER™-ILD clinical study also showed that namilist improved patient survival rates, with a nominally significant reduction in mortality risk.On December 11, InnoCare Pharma announced that Zolotrectinib (ICP-723) had been approved by the NMPA for the treatment of adult and adolescent patients aged 12 years and above with solid tumors harboring NTRK fusion genes. In pivotal registrational clinical trials for patients with NTRK fusion-positive solid tumors, Zolotrectinib demonstrated favorable efficacy and safety as a broad-spectrum anticancer drug without tumor type limitations. The registrational clinical study results showed an overall response rate (ORR) of 89.1%, a disease control rate (DCR) of 96.4%, a 24-month progression-free survival (PFS) rate of 77.4%, and a 24-month overall survival (OS) rate of 90.8%. Zolotrectinib exhibited good overall safety, with data also indicating its ability to overcome resistance to first-generation TRK inhibitors.New Drug Approvals in China (Partial)Progress of Implied Consent for Clinical Trials of New Drugs in ChinaAccording to the PharmaBlock data statistics analysis, the current statistical cycle (2025.12.06-12.12)) A total of 55 new drugs received clinical tacit approval in China, involving 76 application numbers.Among them, there are 17 chemical drugs, 36 therapeutic biological products, and 2 preventive biological products. Compared with the previous statistical cycle, this time there are 6 additional clinical tacit consent acceptance numbers.
This Week's Progress on Clinical Tacit Permits for New Drugs in China (Partial)
Progress in New Drug Applications in ChinaAccording to the data analysis by PharmaDJ, during this statistical period (December 6-12, 2025), there were 13 new drug marketing applications in China, involving 20 acceptance numbers. Among them, there were 5 chemical drugs, 7 therapeutic biologics, and 1 preventive biologic. Compared with the previous statistical period, this period saw an increase of 12 new drug marketing application acceptance numbers.。
New Drug Marketing Applications in China (Partial)According to the data analysis by PharmaBlock, during this statistical period (December 6-12, 2025), a total of 47 new drug clinical trial applications were filed in China, involving 71 application numbers. Among them, there were 23 chemical drugs, 22 therapeutic biological products, and 2 preventive biological products. Compared with the previous statistical period, this period saw a decrease of 17 clinical trial application numbers.
Clinical Trial Applications for New Drugs in China (Partial)According to the PharmaBlock data statistics analysis, the statistical cycle for this period (2025.12.06-12.12)China Adds 4 New ApprovalsNMPADrugs with special qualification designation, including one chemical drug, two biologics, and one cell and gene therapy. Compared to the last statistical cycle, two more drugs received special qualification designation from the NMPA in this round.NMPA Special Qualification Designation DrugsProgress in New Drug Development in ChinaAccording to the data analysis by PharmaDJ, during this statistical period (December 6-12, 2025), there were a total of 16 updates on the clinical R&D status of new drugs in China, covering six fields including neurological diseases, nutritional and metabolic diseases, infectious diseases, and immune system diseases. Among them, there were 11 updates for chemical drugs, 3 for biologics, and 2 for cell and gene therapies. Compared with the previous statistical period, this period saw an increase of four updates on the clinical R&D status of new drugs in China.
On December 7, Ascentage Pharma announced that it had orally presented the latest results of a registrational Phase II clinical study in China on its Bcl-2 inhibitor, lisocabtagene maraleucel, as a monotherapy for relapsed/refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) patients who failed treatment with Bruton's tyrosine kinase inhibitors (BTKi), at the 67th American Society of Hematology Annual Meeting.The research data presented in this oral report indicate that lisocellimab monotherapy demonstrated significant and durable efficacy in heavily pretreated R/R CLL/SLL patients resistant to BTK inhibitors, with manageable safety and no observed cases of tumor lysis syndrome (TLS). The study enrolled a total of 77 patients, among whom 33 (42.9%) had complex karyotypes, 30 (39%) carried del(17p)/TP53 mutations, and 41 (53.2%) exhibited unmutated IGHV. The prevalence of these high-risk factors was significantly higher than in previous similar studies. Notably, among patients with complex karyotypes, 63.6% also harbored del(17p)/TP53 mutations, and 63.6% were identified as having highly complex karyotypes (>=5 chromosomal abnormalities). The objective response rate (ORR) for lisocellimab in all evaluable patients reached 62.5%. Overall, the study results showed that lisocellimab monotherapy achieved encouraging depth and sustained responses in heavily pretreated patients, particularly those with extremely high-risk features such as complex karyotypes.On December 8, CARsgen Therapeutics announced that the clinical data of its off-the-shelf BCMA CAR-T product CT0596 for the treatment of relapsed/refractory multiple myeloma (R/R MM) was presented as a poster at the 67th American Society of Hematology (ASH) Annual Meeting. This clinical trial (NCT06718270) enrolled a total of 8 R/R MM patients in the dose-escalation phase to receive CT0596 infusion.As of August 31, 2025, all eight infused patients were evaluable for efficacy, with a median follow-up time of 4.14 months. Six patients achieved partial response (PR) or better: three achieved complete response/strict complete response (CR/sCR) (all belonging to the full-dose lymphodepletion group), one achieved very good partial response (VGPR), and two achieved PR. Among the six patients who received full-dose lymphodepletion conditioning, five achieved PR or better. All six patients in the full-dose lymphodepletion group tested negative for minimal residual disease (MRD) by the fourth week of treatment. Patient 01 continued to achieve sCR and remained MRD-negative as of the eighth month. Patient 04 achieved PR after the second infusion, with extramedullary disease showing resolution. CAR-T cell expansion was observed in all eight patients. Among the two patients receiving a dose of 4.5×10⁸, one achieved sCR and the other showed deepening efficacy to VGPR. CT0596 demonstrated controllable safety, with no dose-limiting toxicity, treatment interruption, or death events observed. Currently, the study remains in the dose-exploration phase, with the lymphodepletion dose confirmed and higher cell doses being explored to further determine the recommended cell dose (RD).Progress in New Drug Development in China (Partial)
Policy and Regulatory Updates in China's New Drug Development FieldNo relevant policies or regulations were released this week.Hot News in China's New Drug Development FieldAscentage Pharma's "Trump Card"Recently, Ascentage Pharma has once again announced good news in the development of its core product, Nulrex® (Olverembatinib) —— the research progress of this drug in the field of gastrointestinal stromal tumors has been published in the international authoritative journal *Signal Transduction and Targeted Therapy*.At the same time, Ascentage Pharma's performance in the capital market is equally remarkable. Since its successful listing on NASDAQ on January 24, 2025, becoming the first U.S.-listed biopharmaceutical stock of the year, the company's Hong Kong stock price has doubled within the year, reaching a new high since its IPO.What does Ascentage Pharma rely on to win market favor amid the ongoing capital chill in the biopharmaceutical industry? What exactly is its consistently upward "ace"?More information, read the original textNucleic Acid Targeted Therapy Advances FurtherIn November this year, Arrowhead Pharmaceuticals' new drug Plozasiran Injection was approved by the FDA for marketing as an adjunct to diet for reducing triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). Plozasiran Injection is currently the world’s first APOC3 siRNA drug for treating FCS, which lowers triglyceride levels by inhibiting the production of apolipoprotein C-III (APOC3).More information, read the original textLittle D Has Something to Say
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